Automation, digitalization, and supply-chain strategies help mitigate vulnerabilities.

Cell Therapy Manufacturing Amid Pandemic 

EUA for Lilly's mAb treatment; Pifzer reports 90% vaccine efficacy in interim results

COVID-19 Treatments and Vaccines Update

On Nov. 23, 2020, ARCA biopharma, a US-based biopharmaceutical company specializing in a precision-medicine approach to developing cardiovascular therapeutics, announced that FDA has granted fast track designation for its investigational compound, AB201, a small recombinant fusion protein, for the potential treatment of COVID-19.

The company plans to initiate a Phase IIb clinical trial in December 2020 of the compound in approximately 100 patients hospitalized with COVID-19, with topline trial data anticipated in the second quarter of 2021. AB201 is a potent, selective inhibitor of tissue factor (TF), which plays a central role in the inflammatory response to viral infections.

“Fast [t]rack designation for the AB201 development program is an important acknowledgement of the critical need for treatments for hospitalized COVID-19 patients, whether effective vaccines are approved and available or not,” said Dr. Michael Bristow, ARCA’s president and CEO, in a company press release. “We believe AB201’s combination of anticoagulant, anti-inflammatory, and antiviral effects may favorably impact clinical recovery of patients hospitalized with COVID-19 and look forward to beginning the ASPEN-COVID-19 trial to evaluate AB201’s potential efficacy in this patient population.”

Articles

FDA granted fast track designation for AB201, a small recombinant protein that is a potent, selective inhibitor of tissue factor, for the potential treatment of COVID-19. 

FDA Grants Fast Track Designation to ARCA biopharma’s Potential COVID-19 Treatment

Bristol Myers Squibb announced on Nov. 24, 2020 that the European Commission (EC) approved Opdivo (nivolumab), a programmed death-1 immune checkpoint inhibitor, for the second-line treatment of unresectable advanced, recurrent, or metastatic esophageal squamous cell carcinoma (ESCC) in adults who have had prior fluoropyrimidine- and platinum-based combination chemotherapy.

The EC’s decision is based on results from a Phase III study (sponsored by Ono Pharmaceutical, Japan) that demonstrated a statistically significant and clinically meaningful improvement in overall survival in patients who received Opdivo versus chemotherapy. The safety profile for Opdivo was favorable compared with chemotherapy and consistent with previously reported studies of Opdivo in other solid tumors, Bristol Myers Squibb reported in a company press release.

“Today’s approval marks a critically important milestone for those living with esophageal squamous cell carcinoma, as this is the first time an immunotherapy treatment option has been approved in the European Union for this patient population,” said Ian M. Waxman, MD, development lead, gastrointestinal cancers, Bristol Myers Squibb, in the press release. “We are proud of our work in advancing treatment options for people living with upper gastrointestinal cancers, and we look forward to working with European stakeholders to bring Opdivo to more eligible patients who may benefit.”

In addition to this approval in the European Union, Opdivo has been approved in five countries, including the United States and Japan, for the second-line treatment of patients with unresectable advanced, recurrent or metastatic ESCC.

The EC’s approval was based on positive results from a Phase III clinical trial that demonstrated a statistically significant and clinically meaningful improvement in patients compared to chemotherapy. 

EC Approves Bristol Myers Squibb’s Opdivo in Second-Line Treatment of Esophageal Cancer

On Nov. 23, 2020, biopharmaceutical company CureVac and contract development and manufacturing organization Wacker Chemie jointly announced that they have signed a contract for the manufacture of CureVac’s COVID-19 mRNA-based vaccine candidate, CVnCoV. Under terms of the initial agreement, Wacker will ramp up good manufacturing practice (GMP) production of the mRNA drug substance for the vaccine at its biotech site in Amsterdam in the first half of 2021.

Preparations for the start of production, technology transfers, and test runs are already underway, the companies reported in a press release. Wacker plans to produce more than 100 million doses of the CureVac vaccine per year at its Amsterdam site. The site can also be further expanded to meet rising demand in the future.

The Amsterdam site has a history of producing vaccines for clinical development and commercial supply, with a portfolio that ranges from conventional live and killed vaccines to protein-based, polysaccharide and glycoconjugate vaccines. Wacker has invested in the site in recent months to extend production to include mRNA-based vaccines.

Wacker will support production of CureVac’s COVID-19 mRNA-based vaccine candidate at its biotech site in Amsterdam, with production scheduled to start in the first half of 2021.

CureVac and Wacker Sign Manufacturing Contract for COVID-19 Vaccine Candidate

Dolomite Microfluidics, a UK-based microfluidic solutions provider, has partnered with MilliporeSigma to create a range of off-the-shelf NanoFabTx microfluidic device kits that can produce poly (lactic-co-glycolic acid) (PLGA) nano- and microparticles for drug development and controlled drug release applications. The kits (available exclusively from MilliporeSigma) can also be applied to the encapsulation of drugs and other therapeutics in liposomes, such as the SARS-CoV-2 (COVID-19) vaccines currently under investigation.

The increasing use of biodegradable PLGA polymers for drug encapsulation and formulation of controlled release preparations has created a market for easy-to-use, ready-to-go microfluidic solutions. These solutions simplify the workflow for researchers, Dolomite stated in a Nov. 10, 2020 press release.

The new NanoFabTx kits include a microfluidic chip, holders, and accessories, along with application data for the production of a variety of particle types and sizes. To use the kit, simply connect it to the pumps and start the application, Dolomite noted in its press release. This microfluidic approach is user-friendly and offers better encapsulation efficiency and higher monodispersity than traditional methods.

The kit offers the benefit of particle isolation (for particles of the correct dimensions) without wasteful filtering, which ensures the tight control of particle size and shape that is essential to regulating the speed of drug delivery and release. The new kits are intended for use with MilliporeSigma’s NanoFabTx PLGA-Nano and NanoFabTx PLGA-Micro Reagent kits and are optimized for microfluidic pumps and software from Dolomite, to give high accuracy and consistency. The kits can also be combined with Dolomite’s high throughput Telos technology for scaled up experiments.

“MilliporeSigma chose Dolomite Microfluidics because of our reputation and many years’ expertise in providing excellent microfluidic solutions that deliver consistent and reliable production of particles. The controllability and reproducibility that microfluidics offers means the process is really finding its place in drug and vaccine development, making it the method of choice. We are looking forward to continuing our work with scientists to keep getting particles out there at the forefront of this area,” said Richard Gray, commercial director at Dolomite Microfluidics, in the press release.

Dolomite Microfluidics and MilliporeSigma have begun a collaboration to release off-the-shelf microfluidic device kits for the fabrication of PLGA particles.

Dolomite Microfluidics Partners with MilliporeSigma for Microfluidic Device Kits 

The recent commercial launch in November 2020 of a new, small, portable microplate reader by Enzo Biochem, a US-based biosciences and diagnostics company, is expected to simplify laboratory workflows. The new microplate reader can be used with Enzo’s immunoassays and, ultimately, molecular diagnostics, providing new opportunities in point-of-care medicine.

The advanced design and technology of the compact microplate reader makes it possible to deliver precise and accurate results. Used together with Enzo’s proprietary software, the device can be used in a wide variety of applications based on the miniaturization of Enzo’s assays, including metabolites, hormones (e.g., pregnancy), proteins (e.g., inflammatory cytokines), and protein characterization.

The instrument provides researchers and lab professionals with a dedicated, personal device to conveniently fit their space and needs and offers accessibility not previously available. The handheld instrument is about one-tenth the size of the average benchtop plate reader, allowing it to fit into any laboratory setting and saving bench space. At the same time, the device provides new levels of flexibility and portability that can be extended to point-of-care facilities, allowing physicians to provide molecular testing, including assays for viral detection and sexually transmitted diseases.

With this new technology, point-of-care testing can move from centralized, complex clinical labs closer to patient care, Enzo stated in a Nov. 5, 2020 company press release. The technology simplifies testing procedures and opens up the opportunity to shift testing out of the hands of licensed medical technicians to healthcare professionals operating nearer to patients or, in some instances, even to patients themselves.

“Access to point-of-care testing continues to grow as technological advances in clinical diagnostic testing drive miniaturization of complex devices. Now, just as computers moved from mainframes to powerful, portable handheld devices, the same is happening within the clinical diagnostic testing industry. This reduced complexity provides high quality and precise testing capabilities to be available to a broader range of healthcare professionals involved in patient management whether they are sitting at an urgent care facility, a doctor’s office or hospital bedside,” said Kara Cannon, chief commercial officer of Enzo, in the press release.

“The point-of-care testing market, currently valued at over $18 [billion] globally, has the potential to aggressively expand to many testing needs that are now not widely available at point-of-care, including a significant portion of the $15 [billion] immunoassay testing market,” Cannon added. “Common clinical immunoassay tests, such as vitamin D testing and inflammation analysis, currently are performed regularly in the clinical central testing lab by licensed medical technologists using large, complex instrumentation. Unlike lateral flow assays, this small yet powerful system does not sacrifice sensitivity for convenience and allows for multi-assay capabilities.”

“Enzo’s corporate vision addresses new opportunities for expansion and growth within both products and services,” said Cannon in the press release. “We are serving markets including laboratories, point-of-care, and direct-to-consumer. This market launch is an important step in extending our offerings beyond the central laboratory, enabling physicians to provide clinical testing at their office, urgent care facility, or other treatment center.”

Enzo anticipates a future launch of isothermal testing on this same platform, enabling constant temperature and eliminating the need for a thermal cycler.

The new powerful, precise, and compact instrument can be retrofitted for point-of-care clinical testing at urgent care facilities, hospitals, or physicians’ offices.

Enzo’s New Portable Microplate Reader Simplifies Laboratory Workflow

In selecting Domainex, a UK-based biotechnology company specializing in integrated medicines research, to provide integrated lead optimization services, APEIRON Biologics, a privately held Austrian biotech company focused on cancer and respiratory therapies, will be able to advance the development of inhibitors for the E3 ubiquitin ligase Cbl-b using small-molecule drug discovery.

Under this agreement, Domainex will provide its expertise in medicinal and computational chemistry, screening, structural biology, and absorption, distribution, metabolism, excretion, and pharmacokinetics (ADME/PK) to advance APEIRON’s Cbl-b targeting compound series, APN431, towards pre-clinical development.

To date, Domainex has provided APEIRON with its expertise in hit identification, both fragment-based and virtual screening, by establishing and deploying a suite of biophysical assays against variants of the E3 ligase Cbl-b and other members of this target class. These assays allowed the companies to determine the binding affinity, mechanism, and selectivity of promising compounds.

With the expanded deal, the partnership will progress into the next stage, under which Domainex will provide lead optimization services.

“We are delighted to move into this new phase of our partnership with APEIRON Biologics to support the discovery and development of new medicines to treat cancer. Our multi-disciplinary team of scientists has been successful at generating multiple compound series with promising properties. Their commitment through seven-day a week operations and implementation of shift-patterns has enabled us to continue to progress client-sponsored projects throughout the COVID-19 pandemic whilst maintaining social distancing at our state-of the art Medicines Research Centre. We look forward to continuing our journey of discovery with APEIRON,” said Dr. Tom Mander, CEO of Domainex, in a Nov. 9, 2020 company press release.

“Finding the right research partner for the development of our highly innovative immune-oncology therapeutics is essential for fast, efficient, and ultimately successful results. We are delighted to expand our partnership with Domainex for our Cbl-b targeted APN431 project and help us to quickly move to the next stage of its development. Domainex provides deep know-how in state-of-the-art structure based medicinal chemistry and assay development to help us achieve our ambitious goals. With our small-molecule program, APN431, we have an additional approach to target Cbl-b, next to our autologous cell therapy program, APN401, which is currently in clinical Phase I development,” said Peter Llewellyn-Davies, CEO of APEIRON Biologics, in the press release.

The expansion of the companies’ partnership will integrate lead optimization efforts to advance cancer immune therapy drug discovery.

Expansion of APEIRON Biologics and Domainex Partnership will Advance Cancer Drug Discovery

Sanofi announced on Nov. 23, 2020 that the European Commission (EC) approved MenQuadfi (meningococcal [groups A, C, Y, W] conjugate vaccine) for immunization against meningococcal meningitis, the inflammation of the brain and spinal cord caused by a viral infection, in patients from the age of 12 months and older.

The EC’s approval comes after positive results from seven Phase II and III randomized, active-controlled, multi-center studies, a Sanofi press release said. Immunogenicity and safety of the vaccine were assessed in 6300 individuals who received a single dose of MenQuadfi, which showed a good safety profile and induced a high immune response against all four 

“Meningococcal meningitis can take one’s life in as little as one day and leave survivors with severe permanent disabilities. In Europe, there were more than 3000 cases of Invasive Meningococcal Disease in 2018, half of them caused by serogroups C, W, and Y,” said Thomas Triomphe, head of Sanofi Pasteur, in the press release. “One case is one too many. It is our ambition to make this vaccine available worldwide to further expand protection to as many people as possible. The European Commission’s approval of MenQuadfi takes us one step closer to achieving this goal.”

The EC’s approval comes after positive results from seven Phase II and III randomized, active-controlled, multi-center studies.

EC Approves Sanofi’s Meningococcal Meningitis Vaccine 

Mustang Bio, a US-based clinical-stage biopharmaceutical company, and Minaris Regenerative Medicine, a cell and gene therapy contract development and manufacturing organization, announced on Nov. 23, 2020 that they have entered into an agreement for the technology transfer and clinical manufacturing of Mustang’s MB-107 lentiviral gene therapy program for the treatment of X-linked severe combined immunodeficiency (XSCID), also known as bubble boy disease, in Europe.

Through the agreement, Minaris will initiate the technology transfer of the manufacturing and analytical processes and will integrate it into the European regulatory environment for manufacturing at its Ottobrunn, Germany site in order to supply clinical trials in Europe, a Mustang press release said.

“We look forward to a productive and successful partnership with Mustang where Minaris will be able to support them with our extensive experience in the clinical and commercial manufacturing of autologous gene therapies,” said Dusan Kosijer, managing director of Minaris, in the press release. “We are eager to work together with Mustang in the fight against this devastating disease.”

“This agreement with Minaris is an important step in supporting expansion of our MB-107 pivotal clinical trial into Europe,” said Manuel Litchman, MD, president and CEO of Mustang, in the press release. “We look forward to working with Minaris to grow our geographic footprint and bring this potential life-saving therapy to XSCID patients in need internationally.”

The companies have entered into an agreement for the technology transfer and clinical manufacturing of Mustang’s MB-107 lentiviral gene therapy program for the treatment of X-linked severe combined immunodeficiency, also known as bubble boy disease, in Europe.

Mustang Bio to Enter into Agreement with Minaris Regenerative Medicine for Gene Therapy Manufacturing in Europe

Baxter announced on Nov. 24, 2020 that it is investing $50 million into the expansion of its sterile fill/finish manufacturing facilities in Bloomington, IN.

The expansion will include a new 25,000 ft-

 warehouse, a filling line for flexible plastic containers, a high-speed automated syringe fill line with the ability to fill up to 600 units per minute, and a high-speed automated visual inspection line, a company press release said. Construction is expected to be completed in 2021, and the new facilities will create 100 new jobs at the site.

“We pride ourselves on being a contract manufacturing partner with the specialized expertise, proven experience, and facilities to help our clients successfully achieve their sterile manufacturing objectives,” said Marie Keeley, vice-president, BioPharma Solutions, Baxter, in the press release. “Our Bloomington facility is already a global leader in sterile contract manufacturing, and this expansion will add capacity and state-of-the-art technology that will better enable us to meet the diverse needs of our clients and the patients they serve.”

The expansion will include a new 25,000-ft2 warehouse, a filling line for flexible plastic containers, a high-speed automated syringe fill line with the ability to fill up to 600 units per minute, and a high-speed automated visual inspection line.

Baxter to Invest $50 Million into Indiana Fill/Finish Manufacturing Facilities Expansion

Merck, known as MSD outside the United States and Canada, and OncoImmune, a US-based clinical-stage biopharmaceutical company, announced on Nov. 23, 2020 that they have entered into an acquisition agreement where Merck will acquire all outstanding shares of OncoImmune for an upfront payment of $425 million in cash.

Under the terms of the agreement, Merck will fast-track the development of OncoImmune’s COVID-19 therapeutic candidate, CD24Fc, a first-in-class recombinant fusion protein that targets the innate immune system, currently in Phase III clinical development for the treatment of severe and critical disease patients. The therapeutic candidate recently received positive results from an interim efficacy analysis of data from the Phase III study.

“Meaningful new therapeutic options are desperately needed for possibly millions of people around the world who will develop severe or critical COVID-19 disease,” said Dr. Roger M. Perlmutter, president, Merck Research Laboratories, in a company press release. “Recent clinical investigations support the view that CD24Fc may provide benefit beyond standard-of-care therapy for COVID-19 patients requiring oxygen support, and hence will represent an important addition to the Merck pipeline of investigational medicines and vaccines designed to address the COVID-19 pandemic.”

“Outstanding work by the OncoImmune team has provided compelling evidence regarding the use of CD24Fc in patients with severe and critical COVID-19 in our Phase III trial,” added Yang Liu, PhD, co-founder and CEO of OncoImmune, in the press release. “We look forward to working with the scientists and manufacturing engineers at Merck as well as regulators as we seek to accelerate the global development of this potentially important therapy.”

The acquisition gives Merck a new Phase III COVID-19 therapeutic candidate, CD24Fc, a fusion protein that targets the innate immune system, in its pipeline.

Merck to Acquire OncoImmune for $425 Million

Samsung Biologics has officially begun construction on a fourth plant (Plant 4) in Incheon, South Korea. The multi-story, 238,000-m2 biopharmaceutical manufacturing plant will have a total manufacturing capacity of 256,000 L, the company announced in a Nov. 18, 2020 press release.

Upon completion of Plant 4, Samsung Biologics expects to be accountable for a third of the total global biopharmaceutical contract manufacturing organization (CMO) manufacturing capacity with a combined sum of 620,000 L of capacity from a single site. Plant 4 will have a modular design that will allow flexibility for certain parts of the plant to begin manufacturing activities by the end of 2022. Full operations are expected to be on line in 2023.

Equipment and instruments will be fully implemented with data integrity and cybersecurity to reduce risk and to allow real-time secure access to information. The new facility will also be capable of designing to scale the varying needs of clients, from large-scale commercial manufacturing to small-scale CMO. Plant 4’s manufacturing flexibility will be based on a diverse line-up of bioreactors ranging from 2000 L to 10,000 L to 15,000 L. Plant 4 will also provide a full range of contract development organization, CMO, and contract research organization processes as well as early stage development capabilities and large-scale commercial manufacturing with a full quality control lab.

The new super plant, being built in Incheon, South Korea, will have a total biopharmaceutical manufacturing capacity of 256,000 L.

Samsung Biologics Begins Construction on Fourth Biopharma Manufacturing Plant 

Samsung Biologics and Eli Lilly (Lilly) have entered into a long-term manufacturing agreement that is expected to accelerate the global supply for Lilly’s COVID-19 antibody therapies to make these therapies more globally accessible to patients.

Lilly and Samsung Biologics initially entered into a manufacturing partnership in May of 2020 to address the increasingly urgent demand for COVID-19 treatments worldwide. Despite global challenges of securing stable supply of raw materials amid the pandemic, Samsung was able to manufacture and deliver an initial supply of good manufacturing practice-compliant APIs within five months of signing the contract with Lilly. The timeline for tech transfer was also significantly reduced to less than three months because of close communications and coordination between technical, quality, supply chain, and regulatory experts from both companies.

“Neutralizing antibodies are proving to be a potentially important tool in the fight against COVID-19, and the biopharmaceutical industry is collaborating quickly and effectively to increase the global manufacturing supply,” said David A. Ricks, Lilly’s chairman and CEO, in a Nov. 17, 2020 company press release. “This agreement with Samsung supplements Lilly’s own internal manufacturing capacity and greatly expands our ability to deliver Lilly antibody therapies to patients around the world.”

“We are very pleased to deliver this product to our client Lilly in record-breaking speed to aid in the war against the COVID-19 pandemic,” said Dr. Tae Han Kim, CEO of Samsung Biologics, in the press release.

The companies have formed a long-term manufacturing agreement to accelerate the global supply for Lilly’s COVID-19 antibody therapeutics.

Samsung Biologics and Lilly Partner to Accelerate Delivery of COVID-19 Antibody Therapeutics

Samsung Biologics has signed a memorandum of understanding (MOU) with Sartorius to further strengthen the supply chain for biomedicines. The MOU follows a recent announcement by Sartorius that the company plans to invest $100 million in South Korea's bio cluster, Songdo, to build a bioprocess manufacturing facility for equipment and materials, Samsung Biologics announced in a Nov. 16, 2020 press release.

Sartorius plans to supply materials such as disposable bags, cell culture media, and filters to Samsung Biologics. The facility will be built on a 24,333-m2 site in Songdo. Construction is expected to be completed by 2022.

The agreement follows a recent announcement that Sartorius plans to invest $100 million in Songdo, South Korea’s bio cluster.

Samsung Biologics Signs MOU with Sartorius to Enhance Supply Chain for Biomedicines

Roche has signed an option and license agreement with CEVEC Pharmaceuticals to use CEVEC’s recently launched ELEVECTA technology for the large-scale manufacture of adeno-associated virus (AAV) vectors for gene therapy applications. Financial terms of the agreement were not disclosed.

Under the agreement, CEVEC will grant Roche and Spark Therapeutics, a member of the Roche Group, an option for a non-exclusive license to develop AAV producer cell lines based on the ELEVECTA technology. Roche will have the rights to use the technology for its portfolio of gene therapy products while CEVEC will be eligible for payments based on the achievement of certain milestones for products as well as royalties on sales of potential products using the technology, according to a Nov. 10, 2020 press release.

“We are delighted to partner with Roche with the goal to manufacture AAV vector-based gene therapy programs on a large scale by leveraging our novel ELEVECTA Technology. This agreement represents a major milestone while highlighting ELEVECTA as the technology of choice for next generation viral vector manufacturing,” said Dr. Nicole Faust, CEO of CEVEC, in the company press release. “Given the enormous progress of gene therapies, manufacturing technologies must keep up with the needs of the industry in terms of volumes, quality, robustness, and ease of use. With ELEVECTA we are paving the way for many more commercial gene therapy applications in the future.”

“Across the Roche Group, we are always looking for new and innovative technologies that provide clear competitive advantages and add significant value to our portfolio,” said James Sabry, head of Roche Pharma Partnering, in the press release. “We are excited to use CEVEC’s ELEVECTA Technology for potential large-scale production of gene therapies and providing them to patients in need.”

The agreement will allow Roche to use CEVEC’s proprietary ELEVECTA technology, which is designed to enable fully scalable, high-performance AAV vector production in suspension bioprocesses.

Roche in Agreement to Use CEVEC’s ELEVECTA Technology for AAV Manufacture

PathoQuest SAS, a French genomics company specializing in quality control testing of biologics, has established a US subsidiary with its strategic partner, Charles River Laboratories International, a contract research organization (CRO). The new subsidiary is named PathoQuest, Inc.

In addition to the new subsidiary, the partners will also construct a new laboratory at Charles River’s Wayne, PA, site that will use next-generation sequencing (NGS)-based testing technology. The new lab will expand the partners’ capabilities by providing a fully integrated and reliable solution for rapid viral safety testing of biologics, notably advanced therapeutic medicinal products (ATMPs) and vaccines, according to a Nov. 12, 2020 press release.

This expansion will offer North American biotechnology companies an NGS testing facility in the United States that identifies adventitious agents using PathoQuest’s rapid, 

, comprehensive testing approach, accelerating biologic development timelines without compromising safety and efficacy. NGS can also be used to characterize the genetic information of cell lines.

Construction of PathoQuest’s new office and the new lab facility is scheduled to begin in late 2020 with NGS services expected to be available in 2021. Once complete, the lab will align with PathoQuest’s existing lab in Paris and offer current good manufacturing practices-compliant testing.

Charles River’s Wayne facility supports the development of biological products, including cell line characterization, viral safety, and viral clearance studies. Charles River made an additional direct investment in PathoQuest to help fund the new facility and to support the expansion of PathoQuest’s testing platform into North America.

“The addition of a PathoQuest facility at the Wayne site highlights our commitment to addressing our clients’ dynamic needs. PathoQuest’s NGS-based testing solution provides a rapid, reliable, and comprehensive testing solution, and, now, clients will have it at their fingertips through this expanded partnership,” said Birgit Girshick, corporate executive vice-president, Discovery and Safety Assessment, Biologics Testing Solutions, and Avian Vaccine Services, Charles River, in the press release.

“Our NGS-based approach provides a state-of-the-art quality control testing solution for biopharmaceutical quality assurance and production managers. Our new facility in Wayne will create additional opportunities to integrate with Charles River’s service offerings and expose more pharmaceutical and biotechnology groups to our technology, furthering the rapid expansion of our business,” said Jean-Francois Brepson, president and CEO, PathoQuest, in the press release.

The partners have established PathoQuest, Inc., a US subsidiary, and will construct an NGS-based testing lab at Charles River’s Wayne, PA, site. 

Charles River and PathoQuest Establish US Subsidiary for NGS-based Testing Lab 

Jill Wechsler is BioPharm International's Washington Editor, jillwechsler7@gmail.com.

In unveiling a set of drug price control measures last week, the president displayed his bitterness that vaccine makers failed to announce their success in achieving high efficacy rates for new preventives before the Nov. 3 election. Instead of focusing on his administration’s remarkable achievement in financing and supporting record-breaking development of critical vaccines to combat COVID-19, President Trump lashed out at Pfizer for conspiring to delay the good news on its vaccine as part of a conspiracy to block his re-election.

The administration took on Big Pharma in issuing a new final interim rule Nov. 20, 2020 to establish a 

that caps Medicare Part B drug reimbursement for 50 selected medicines at the lowest price paid by any developed country. The policy applies to Medicare Part B drugs, which are administered in doctors’ offices and hospitals and include many costly biotech therapies that require intravenous administration. Although the proposal predicts notable savings for taxpayers, analysts say it is more likely to cut reimbursement for doctors and hospitals. That drew 

 from the American Hospital Association, as well as the Pharmaceutical Research and Manufacturers of America (PhRMA). The most visible savings would go to Medicare, as the rule technically implements a process for testing a new payment method for a federal healthcare program.

The White House also announced that it plans to eliminate drug company rebates to pharmacy benefit managers (PBMs) and insurance companies for Medicare Part D drugs. The idea is to shift those savings to consumers, although a review of an earlier version of this change found that it would raise premiums paid by seniors for Medicare drug coverage and that any savings would go to the government, and not to consumers. A rule to implement this change is under review by the Office of Management & Budget. While manufacturers have applauded curbs on rebates, PBMs and payers are furious and 

third proposal revises federal anti-kickback rules

 on business relationships between doctors and other parties, including biopharma. The changes aim to support a shift toward value-based reimbursement for medical services and products, including drugs. The proposal may clarify policies for when drug company fees to health professionals are appropriate, such as for consulting or participating in speaker programs, or for offering discount cards to cover patient co-pays for certain medicines.

It’s unusual for any administration to release significant new policies so close to the end of a presidential term. The MFN pricing rule was published with just the required 60 days needed to finalize it before Inauguration Day on Jan. 20, 2021. Although Democrats generally support drug pricing limits, it’s not clear how the Biden administration and Congress will respond to the last-minute Trump proposals. In any case, PhRMA says it will consider “all options to stop this unlawful onslaught on medical progress,” and is expected to file suit against the MFN policy.

The Trump administration unveiled a new drug price control policy, drawing opposition from pharma manufacturers and pharmacy benefit managers.

Trump’s Revenge on Pharma

On Nov. 21, 2020, FDA issued an emergency use authorization (EUA) for casirivimab and imdevimab for the treatment of mild to moderate COVID-19 in adults and pediatric patients with positive viral test results who are at high risk of progressing to severe COVID-19.

When administered together in clinical trials, casirivimab and imdevimab were shown to lessen COVID-19 hospitalization or emergency room visits in patients at high risk for disease progression within 28 days after treatment when compared to placebo, an FDA press release said. The monoclonal antibody combination is not permitted for patients who are already hospitalized for COVID-19 or require oxygen therapy because of the disease.

“The FDA remains committed to advancing the nation’s public health during this unprecedented pandemic. Authorizing these monoclonal antibody therapies may help outpatients avoid hospitalization and alleviate the burden on our health care system,” said FDA Commissioner Stephen M. Hahn, MD, in the press release. “As part of our Coronavirus Treatment Acceleration Program, the FDA uses every possible pathway to make new treatments available to patients as quickly as possible while continuing to study the safety and effectiveness of these treatments.”

“The emergency authorization of these monoclonal antibodies administered together offers health care providers another tool in combating the pandemic,” said Patrizia Cavazzoni, MD, acting director of the FDA’s Center for Drug Evaluation and Research, in the press release. “We will continue to facilitate the development, evaluation, and availability of COVID-19 therapies.”

When administered together in clinical trials, casirivimab and imdevimab were shown to lessen COVID-19 hospitalization or emergency room visits in patients at high risk for disease progression within 28 days after treatment when compared to placebo. 

FDA Issues EUA for COVID-19 Monoclonal Antibody Treatment

On Nov. 23, 2020, AstraZeneca announced its COVID-19 vaccine candidate, AZD1222, showed positive high-level results in interim analyses of clinical trials in the United Kingdom and Brazil.

According to a company press release, the vaccine was highly effective in preventing COVID-19, with no hospitalizations or severe cases reported in participants receiving the vaccine. An independent data safety monitoring board determined that the clinical trial data demonstrated that the vaccine candidate met its primary endpoint and demonstrated protection from the disease for 14 days or more after receiving two doses of the vaccine.

Additionally, one dosing of the vaccine showed efficacy of 90% when given as a half dose, followed by a full dose at least one month apart, and another dosing showed 62% efficacy when given as two full doses at least one month apart, the press release said. This resulted in an efficacy of 70%.

“These findings show that we have an effective vaccine that willsavemanylives,” said Professor Andrew Pollard, chief investigator of the Oxford Vaccine Trial at Oxford, in the press release. “Excitingly, we’ve found that one of our dosing regimens may be around 90% effective and if this dosing regime is used, more people could be vaccinated with planned vaccine supply.”

“Today marks an important milestone in our fight against the pandemic. This vaccine’s efficacy and safety confirm that it will be highly effective against COVID-19 and will have an immediate impact on this public health emergency,” added Pascal Soriot, CEO, AstraZeneca, in the press release. “Furthermore, the vaccine’s simple supply chain and our no-profit pledge and commitment to broad, equitable, and timely access means it will be affordable and globally available, supplying hundreds of millions of doses on approval.”

AstraZeneca will now seek regulatory submission of the data to authorities around the world for conditional or early approval. It will also request an Emergency Use Listing from the World Health Organization for vaccine availability in low-income countries.

An independent data safety monitoring board found that clinical trial data demonstrated that the vaccine candidate met its primary endpoint and demonstrated protection from COVID-19.

AstraZenca Vaccine Candidate ‘Highly-Effective’ in Preventing COVID-19

FDA’s Vaccines and Related Biological Products Advisory Committee (VRBPAC) is scheduled to meet on Dec. 10, 2020, to discuss a request from Pfizer and BioNTech Manufacturing GmbH for emergency use authorization (EUA) of BNT162b2, the companies’ COVID-19 vaccine.

The VRBPAC—which includes scientific and public health experts, consumer and industry representatives, and sometimes a patient representative—provides advice to the agency and may provide advice on the safety and effectiveness data submitted in the EUA request. FDA makes the final decision on authorizing a vaccine for emergency.

In terms of timing of the VRBPAC meeting following the submission of the EUA request, this amount of time will allow FDA to thoroughly evaluate the data and information submitted in the EUA request before the meeting and to be prepared for a robust public discussion with the advisory committee

“The FDA recognizes that transparency and dialogue are critical for the public to have confidence in COVID-19 vaccines. I want to assure the American people that the FDA’s process and evaluation of the data for a potential COVID-19 vaccine will be as open and transparent as possible,” said FDA Commissioner Stephen M. Hahn, in a statement. “The FDA has been preparing for the review of EUAs for COVID-19 vaccines for several months and stands ready to do so as soon as an EUA request is submitted.”

Hahn further noted that the while the time needed for FDA review was not certain “FDA will review the request as expeditiously as possible, while still doing so in a thorough and science-based manner, so that we can help make available a vaccine that the American people deserve as soon as possible.”

“A discussion about the safety and effectiveness of Pfizer and BioNTech’s vaccine with this committee, made up of outside scientific and public health experts from around the country, will help ensure clear public understanding of the scientific data and information that the FDA will evaluate in order to make a decision about whether to authorize a vaccine for emergency use for the prevention of COVID-19,” Hahn noted in the statement.

The agency will make background materials available to the public, including the meeting agenda and committee roster, no later than two business days prior to the meeting, FDA reported.

In the Nov. 20, 2020 statement, FDA reported the agency’s intent to share details of the meeting in the 

during the week of Nov. 23, including information about a public docket for submission of comments. At that time, public comments can be submitted. The agency said the meeting will be livestreamed on the agency’s YouTube, Facebook, and Twitter channels; and will be webcast from the FDA website.

Following an EUA application from Pfizer and NioNTech, FDA outlines the process for committee and agency review. 

FDA Schedules COVID-19 Vaccine Review for Dec. 10. 2020

ApiJect Systems, a public benefit corporation based in Connecticut, announced on Nov. 19, 2020 that it has been approved by the US International Development Finance Corporation (DFC) for a $590 million loan to construct a multi-facility campus for fill/finish of injectable medicines and vaccines in the event of a national emergency, beginning with COVID-19.

 campus will be located in Research Triangle Park, NC. The company intends to build a facility, which it is calling the Gigafactory, to package up to three billion single-dose prefilled injectors annually using blow-fill-seal (BFS) technology using ApiJect's proprietary pen needle-style hubs. The factory will be Biosafety Level 2 (BSL-2) capable and support vaccine candidates that require standard cold storage or ultra-cold storage down to -70 °C. By isolating each manufacturing line, up to 15 different drugs can be packaged simultaneously.

The new ApiJect campus will also include two drug manufacturing facilities to handle drugs such as the next generation of antibiotics and cytotoxic drugs. The campus will also have a needle and cannula factory.

ApiJect currently has a BFS facility, with a partner company, The Ritedose Corporation (TRC), based in Columbia, SC. As part of the US Operation Warp Speed, ApiJect repurposed and upgraded the manufacturing facility at TRC to have the capacity to aseptically fill and finish up to 45 million doses per month of vaccines and other injectable medicines.

"The DFC loan enables us to transition as quickly as possible from the emergency short-term program of 45 million a month of dose capacity created under Operation Warp Speed to the Gigafactory's projected capacity of 250 million doses a month,” said Franco Negron, CEO of ApiJect. “This project will ensure America is never caught short in its ability to fill and finish vaccines and injectable medicines necessary to respond to population-wide health threats ranging from COVID-19 to any potential future bio-emergencies."

According to the press release, the US government has the right to reserve as many of the Gigafactory's lines as may be necessary to respond to any national health emergency. The facility will also fill and finish other critical life-saving injectable medicines and vaccines for commercial pharmaceutical clients when not devoted to providing products for national health security.

Apiject was approved for a $590-million loan from the US to build in-country capacity for COVID-19 vaccines and therapeutics. 

Featured Content

Cell Therapy Manufacturing Amid Pandemic

COVID-19 Treatments and Vaccines Update